Western Australian adolescent emotional wellbeing during the COVID-19 pandemic in 2020

Background The impacts of the COVID-19 pandemic have been vast and are not limited to physical health. Many adolescents have experienced disruptions to daily life, including changes in their school routine and family’s financial or emotional security, potentially impacting their emotional wellbeing. In low COVID-19 prevalence settings, the impact of isolation has been mitigated for most young people through continued face-to-face schooling, yet there may still be significant impacts on their wellbeing that could be attributed to the pandemic. Methods We report on data from 32,849 surveys from Year 7–12 students in 40 schools over two 2020 survey cycles (June/July: 19,240; October: 13,609), drawn from a study of 79 primary and secondary schools across Western Australia, Australia. The Child Health Utility Index (CHU9D) was used to measure difficulties and distress in responding secondary school students only. Using comparable Australian data collected six years prior to the pandemic, the CHU9D was calibrated against the Kessler-10 to establish a reliable threshold for CHU9D-rated distress. Results Compared to 14% of responding 12–18-year-olds in 2013/2014, in both 2020 survey cycles almost 40% of secondary students returned a CHU9D score above a threshold indicative of elevated difficulties and distress. Student distress increased significantly between June and October 2020. Female students, those in older Grades, those with few friendships or perceived poor quality friendships, and those with poor connectedness to school were more likely to score above the threshold. Conclusions In a large dataset collected during the first year of the COVID-19 pandemic, the proportion of secondary school students with scores indicative of difficulties and distress was substantially higher than a 2013/2014 benchmark, and distress increased as the pandemic progressed, despite the low local prevalence of COVID-19. This may indicate a general decline in social and emotional wellbeing exacerbated by the events of the pandemic. Trial registration: ANZCTRN (ACTRN12620000922976). Retrospectively registered 17/08/2020. https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=380429&isReview=true.

Trends in Partial Disability-Free Life Expectancy between the Ages of 45 and 70 in Canada, 2000–2014

To better evaluate the benefits of a possible increase in the normal retirement age, this article proposes to examine recent trends in the health status of Canadians between 45 and 70 years of age. Using the Sullivan method, trends from 2000 to 2014 in partial disability-free life expectancy (PDFLE) between the ages of 45 and 70 years are computed. Disability is estimated using attributes of the Health Utility Index correlated with the capacity to work, and is looked at by level of severity. Data from the Canadian Community Health Survey were used to estimate the prevalence of disability. Results reveal a slight increase in partial life expectancy between the ages of 45 and 70, and a larger number of those years spent in poor health since the beginning of the 2000s. Hence, this study brings no evidence in support of the postponement of the normal retirement age if this policy were solely based on gains in life expectancy.

Updated Health-Related Quality of Life Results from the KarMMa Clinical Study in Patients with Relapsed and Refractory Multiple Myeloma Treated with the B-Cell Maturation Antigen-Directed Chimeric Antigen Receptor T Cell Therapy Idecabtagene Vicleucel (ide-cel, bb2121)

Introduction: Patients with heavily pretreated relapsed and refractory multiple myeloma (RRMM) have poor outcomes and poor health-related quality of life (HRQoL). The B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell therapy ide-cel has shown frequent, deep, and durable responses in patients with RRMM who were triple-class exposed (TCE) to immunomodulatory drugs, proteasome inhibitors (PI), and anti-CD38 monoclonal antibodies in the pivotal, phase 2, single-arm KarMMa trial (Munshi NC, et al. N Engl J Med 2021;384:705-716). Ide-cel is approved in the US by the FDA for the treatment of adults with RRMM after ≥ 4 prior lines of therapy, including an immunomodulatory drug, PI, and anti-CD38 monoclonal antibody. We have shown previously that in the KarMMa trial, ide-cel provides clinically meaningful benefits in HRQoL at 9 months' follow-up (Delforge M, et al. HemaSphere 2020;4(suppl 1). Abstract EP1000; Shah N, et al. Blood 2020;136(suppl 1):28-29). The aim of this analysis was to extend previous reports by analyzing the effect of ide-cel on HRQoL at 24 months post-infusion data cut off (December 21, 2020) in patients with TCE RRMM in the KarMMa trial. Methods: In the KarMMa trial (NCT03361748), eligible patients had ≥ 3 prior antimyeloma treatment regimens, were TCE, and refractory to their last treatment regimen per International Myeloma Working Group criteria. To assess HRQoL, European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ) C30 (QLQ-C30), EORTC QLQ Multiple Myeloma Module (QLQ-MY20) and EuroQoL 5 dimensions 5 levels (EQ-5D-5L) questionnaires were administered at screening, baseline, at ide-cel infusion, at months 1-6, and every 3-6 months up to month 24 or end of study. Thresholds for clinically meaningful changes from baseline were predefined. Statistical significance was calculated using the 2-sided Wilcoxon signed rank test (0.05 significance level). Results: Of 128 patients treated with ide-cel, 126 (98%) had a baseline and ≥ 1 post-baseline HRQoL assessment and were included in the HRQoL-evaluable population. The patient questionnaire completion rate was ≥ 75% up to month 6 and 50%-70% thereafter for most visits. For the predefined primary HRQoL domains, mean scores improved after ide-cel treatment and were comparable to the general population (Table). Mean changes from baseline exceeded the minimal important difference (MID) threshold for clinically meaningful improvement in fatigue, pain, physical functioning, cognitive functioning, and global health status/quality of life (QoL) scores of QLQ-C30 and disease symptom scores of QLQ-MY20 through month 24 (data cutoff, December 21, 2020). Side effects of treatment (QLQ-MY20) remained stable. Overall, 40%-70% of patients had clinically meaningful improvements in the QLQ-C30 fatigue, pain, physical functioning, and global health status/QoL scores at later timepoints. Moreover, 30%-40% of patients experienced clinically meaningful improvements in cognitive functioning, disease symptoms, and side effects, with 40%-60% of patients remaining stable in these domains, across most of the post-baseline assessment visits. Predefined secondary HRQoL domains included all other domains from QLQ-C30 and -MY20, EQ-5D-5L health utility index scores, and EQ-5D visual analogue scale (VAS) scores. Mean changes from baseline exceeded the MID thresholds for clinically meaningful improvement and reached statistical significance across most follow-up visits for role functioning, emotional functioning, social functioning, dyspnea, insomnia, constipation, diarrhea (QLQ-C30), future perspectives (QLQ-MY20), health utility index scores (EQ-5D-5L), and VAS scores (EQ-5D). Mean changes from baseline for nausea/vomiting, appetite loss, financial difficulties (QLQ-C30), and body image (QLQ-MY20) scores were not clinically meaningful. At the individual level, most patients remained stable or achieved clinically meaningful improvements in secondary domains of interest across almost all follow-up visits. Conclusion: In this study, patients with TCE RRMM who received a single infusion of ide-cel showed clinically meaningful improvements across multiple HRQoL domains during the 24-month follow-up period. Figure 1 Figure 1. Disclosures Delforge: Bristol Myers Squibb: Honoraria, Membership on an entity's Board of Directors or advisory committees; Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees. Shah: Sanofi: Consultancy; Sutro Biopharma: Research Funding; Janssen: Research Funding; Oncopeptides: Consultancy; Kite: Consultancy; Indapta Therapeutics: Consultancy; Poseida: Research Funding; Nektar: Research Funding; Precision Biosciences: Research Funding; Karyopharm: Consultancy; GSK: Consultancy; CareDx: Consultancy; BMS/Celgene: Research Funding; Bluebird Bio: Research Funding; CSL Behring: Consultancy; Amgen: Consultancy; Teneobio: Research Funding. Rodríguez-Otero: Sanofi: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; GSK: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Oncopeptides: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Janssen: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; BMS/Celgene: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Takeda: Consultancy; Janssen, Celgene, Amgen, Oncopeptides, Sanofi, Abbvie, GlaxoSmithKline, Kite Pharma: Consultancy, Honoraria, Speakers Bureau; Regeneron: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; Amgen: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau. Braverman: BMS: Current Employment, Current equity holder in publicly-traded company. Dhanda: BMS: Current Employment, Current equity holder in publicly-traded company. Shi: Bristol Myers Squibb: Consultancy. Guo: Bristol Myers Squibb: Consultancy; Daiichi Sankyo: Consultancy; UCB: Consultancy; Janssen: Consultancy; Gilead: Consultancy; EMD Serono: Consultancy; Evidera: Current Employment. Yu: Evidera: Current Employment. Liao: Evidera: Current Employment. Campbell: Bristol Myers Squibb: Current Employment, Current holder of individual stocks in a privately-held company. Munshi: Legend: Consultancy; Celgene: Consultancy; Karyopharm: Consultancy; Abbvie: Consultancy; Pfizer: Consultancy; Adaptive Biotechnology: Consultancy; Novartis: Consultancy; Oncopep: Consultancy, Current equity holder in publicly-traded company, Other: scientific founder, Patents & Royalties; Takeda: Consultancy; Amgen: Consultancy; Janssen: Consultancy; Bristol-Myers Squibb: Consultancy.

Health-Related Quality of Life in Multiple System Atrophy using EQ-5D-5L: A Large Cross-Sectional Study in China

Background Multiple system atrophy (MSA) is a rare neurodegenerative disease, featuring autonomic failure plus parkinsonism and/or cerebellar ataxia. These symptoms impact the health-related quality of life (HRQoL) of MSA. Objective We aimed to evaluate the HRQoL of MSA with a preference-based instrument, the five-level EuroQol five-dimensions questionnaire (EQ-5D-5L), for the first time.Methods EQ-5D-5L was used to evaluate the HRQoL. The result of HRQoL was displayed as heath utility index and visual analog scale (EQ-VAS) score. Specific scales were used to measure the disease severity, cognition, frontal lobe function, anxiety, depression, fatigue, and sleep disorders. The forward logistic model was used to explore the determinants of HRQoL in MSA.Results A total of 205 patients with cerebellar variant (MSA-C, 53.9%) and 175 patients with parkinsonian variant (MSA-P, 46.1%) patients were included in the study. The mean scores of the health utility index and EQ-VAS were 0.558 and 59.5, respectively. Mobility was reported by the largest proportion (92.1%) of MSA patients, followed by usual activities (88.7%), self-care (81.3%), anxiety/depression (72.1%), and pain/discomfort (53.9%). The determinants of the lower health utility index in MSA were female sex, greater total Unified Multiple System Atrophy Rating Scale (UMSARS) scores, fatigue, and Parkinson's disease-related sleep problems (PD-SP). Lower EQ-VAS score was associated with greater total UMSARS scores, fatigue, PD-SP, and anxiety symptom. MSA-P patients reported more frequent problems in pain/discomfort than MSA-C patients, while MSA-C patients reported more problems in mobility than MSA-P patients. Conclusion Patients with MSA had poor HRQoL evaluated by EQ-5D-5L. The most frequent affected problem is mobility in the Chinese MSA population. Besides the severity of MSA, fatigue, PD-SP and anxiety were determinants for poor HRQoL. Our research provides important information to improve the health status of patients with MSA.

Health-related quality of life in multiple system atrophy using EQ-5D-5L: a large cross-sectional study in China

Background Multiple system atrophy (MSA) is a rare neurodegenerative disease, featuring autonomic failure plus parkinsonism and/or cerebellar ataxia. These symptoms impact the health-related quality of life (HRQoL) of MSA. Objective We aimed to evaluate the HRQoL of MSA with a preference-based instrument, the five-level EuroQol five-dimensions questionnaire (EQ-5D-5L), for the first time. Methods EQ-5D-5L was used to evaluate the HRQoL. The result of HRQoL was displayed as heath utility index and visual analog scale (EQ-VAS) score. Specific scales were used to measure the disease severity, cognition, frontal lobe function, anxiety, depression, fatigue, and sleep disorders. The forward logistic model was used to explore the determinants of HRQoL in MSA. Results A total of 205 patients with cerebellar variant (MSA-C, 53.9%) and 175 patients with parkinsonian variant (MSA-P, 46.1%) patients were included in the study. The mean scores of the health utility index and EQ-VAS were 0.558 and 59.5, respectively. Mobility was reported by the largest proportion (92.1%) of MSA patients, followed by usual activities (88.7%), self-care (81.3%), anxiety/depression (72.1%), and pain/discomfort (53.9%). The determinants of the lower health utility index in MSA were female sex, greater total Unified Multiple System Atrophy Rating Scale (UMSARS) scores, fatigue, and Parkinson's disease-related sleep problems (PD-SP). Lower EQ-VAS score was associated with greater total UMSARS scores, fatigue, PD-SP, and anxiety symptom. MSA-P patients reported more frequent problems in pain/discomfort than MSA-C patients, while MSA-C patients reported more problems in mobility than MSA-P patients. Conclusion Patients with MSA had poor HRQoL evaluated by EQ-5D-5L. The most frequent affected problem is mobility in the Chinese MSA population. Besides the severity of MSA, fatigue, PD-SP and anxiety were determinants for poor HRQoL. Our research provides important information to improve the health status of patients with MSA.

The Benefit of Endovascular Thrombectomy for Stroke on Functional Outcome Is Sustained at 12 Months

<b><i>Background and Purpose:</i></b> The short-term benefits of endovascular thrombectomy (EVT) for acute ischemic stroke (AIS) have been widely documented, yet there is limited evidence to show that this is sustained in the long term. We aimed to determine whether the benefit of EVT on functional outcome at 3 months is maintained at 12 months and the factors correlating with functional independence and quality of life. <b><i>Methods:</i></b> Data for analysis came from a prospective registry of consecutive patients undergoing EVT at a single Comprehensive Stroke Center (Oct 2018–Sep 2019). A phone interview was conducted for 12-month patient outcomes. Functional outcome was assessed by the modified Rankin Scale (mRS). Quality of life was determined by return to usual place of residence, work, or driving and calculation of a health utility index using the European Quality of Life-5 Dimensions questionnaire (EQ-5D-3L). <b><i>Results:</i></b> Of the 151 patients who underwent EVT during the study period, 12-month follow-up was available for 145 (96%). At 12 months, 44% (<i>n</i> = 64) of patients were functionally independent (mRS 0–2) compared to 48% at 3 months. Mortality at 12 months was 26% compared to 17% at 3 months. Significant predictors of functional independence at 12 months were younger age and lower baseline National Institutes of Health Stroke Scale. Better quality of life significantly correlated with return to usual place of residence and driving. <b><i>Conclusion:</i></b> Three-month functional independence was sustained at 12 months, indicating that EVT remains beneficial for patients with AIS in the longer term.

An Assessment of the Validity and Reliability of the Pediatric Child Health Utility 9D in Children with Inflammatory Bowel Disease

Health utilities relevant to children are lacking, compromising health funding and policy decisions for children. The Child Health Utility 9D (CHU9D) is a recently developed preference-based health utility instrument designed for use in children. The objective was to examine the validity of the CHU9D in a cohort of 285 Canadian children aged 6.5 to 18 years of age with Crohn’s disease (CD) and ulcerative colitis (UC), (collectively inflammatory bowel disease (IBD)). The correlation and agreement between paired CHU9D and Health Utility Index (HUI) assessments were determined with Spearman coefficients and Bland–Altman levels of agreement. Total and domain utilities were calculated for the CHU9D using Australian adult and youth tariffs. Algorithms for HUI2 and HUI3 were used. Domain correlations were determined between domains with expected overlap between instruments. In CD and in UC, correlations between CHU9D, HUI2, and HUI3 utilities ranged between 0.62 to 0.67 and 0.67 to 0.69, respectively (p < 0.05). CHU9D utilities were lower using youth tariffs compared to adult tariffs. A large range in health utilities suggested a heterogeneous quality of life. The CHU9D is a good option for preference-based utility measurement in pediatric IBD. Additional research is required to derive pediatric tariffs to conduct economic evaluation in children.

Burden of Spinal Muscular Atrophy (SMA) on Patients and Caregivers in Canada

Background: Spinal muscular atrophy (SMA) is a rare neurodegenerative disease characterized by progressive muscular weakness, which occurs in one in 6,000 to 10,000 live births. The burden of SMA on Canadian patients and caregivers is not known. Objective: To characterize the burden of SMA in Canada as reported by patients and caregivers, including disease and treatment impacts, indirect costs, and caregiver burden. Methods: Surveys were distributed by Cure SMA Canada and Muscular Dystrophy Canada to individuals with SMA and their caregivers. The online surveys were anonymous and completed between January 28 and February 21, 2020. Results: 965 patient and 962 caregiver responses met the eligibility criteria. Patients reported SMA subtypes as: type I (25.0%), type II (41.3%), type III (29.3%). Using the EQ-5D, patients were shown to have impaired quality of life with an average health utility index of 0.49 (SD: 0.26). The median expenditure was $4,500 CAD (IQR: $1,587 – $11,000) for assistive devices; $6,800 CAD ($3,900–$13,000) on health professional services; and $1,200 CAD (IQR: $600 –$3,100) on SMA-related travel and accommodation in the past 12 months. Caregivers reported needing respite care (45.7%), physiotherapy for an injury from a lift/transfer (45.7%), or other health impacts (63.3%). Caregivers reported changes to personal plans, sleep disturbances, and work adjustments, with a mean Caregiver Strain Index score of 7.5 [SD: 3.3]. Conclusion: SMA in Canada is associated with a significant burden for patients and their caregivers.

Abstract P259: The Association Between Sex, Gender and Health Status of Stroke Survivors in the Canadian Population

Background: Stroke is one of the most common cerebrovascular diseases causing permanent disability, and decreased quality of life (QoL). Both sex and gender have been reported to be associated with health outcomes. Gender, unlike biological sex, encompasses the psycho-socio-cultural roles, behaviors and identities of men, women, and gender-diverse people. Hypothesis: To examine the association between sociocultural gender, biological sex and health status among stroke survivors in the Canadian population. Methods: Data from cycles 2013-2014 and 2015-16 (n=237,121) of the Canadian Community Health Survey (CCHS) were analyzed. The primary endpoint of the study was Health Utility Index (HUI), a measure of health status and QoL. This index measures a range of health domains (i.e. vision, hearing, speech, ambulation, dexterity, emotion, cognition, and pain) and ranges between -0.36 (severe health state) to 1 (perfect health state). A gender score was computed based on the Genesis-Praxy method, using a principal component analysis-derived propensity score method. The final gender scores ranging from 0 to 1 (higher score identifying characteristics traditionally ascribed to women) included household size, perceived life stress, education, sense of belonging to community, marital status, and income. All statistical analyses were performed using R (V.4.0.2) with survey design. Results: Amongst 3,773 (1.1%) stroke survivors in two cycles, 47.8% were female and a majority were older than 50 years (85.3%). Overall, 76.4% of the stroke survivors had moderate to severe HUI (<=0.88), however, this rate was higher in females (82.5% vs 70.2%, P<0.001). Median gender score was 0.49 [0.46-0.55]. Higher gender scores (OR=12.5, 95%CI=1.4-116.2, P=0.02) and female sex (OR=1.8, 95%CI=1.2-2.8, P=0.002) were independently associated with moderately to severely diminished health status (HUI) in a model adjusted for age, and comorbidities (i.e. hypertension, diabetes, heart disease, and history of cancer). Conclusion: Characteristics traditionally ascribed to women’s gender and female sex were associated with poorer health status in stroke survivors. Gender-related factors must be targeted for improving the health status of patients suffering from stroke.